RECOMBINANT ADEN-ASSOCIATED VIRUS, WHICH IS HYBRID OF AAV9 AND AAVrh74 SEROTYPES, WITH REDUCED TROPISM FOR LIVER TISSUE Russian patent published in 2024 - IPC C12N15/864 A61K48/00 

Abstract RU 2812279 C2

FIELD: biotechnology.

SUBSTANCE: recombinant adeno-associated virus (AAV) capsid protein is described that is a hybrid of the capsid proteins of AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74), comprising a sequence having at least 85% identity with the following SEQ ID NO: 3, and has a reduced tropism for liver tissue compared to the capsid proteins of the original serotypes AAV9 and AAVrh74. The following is presented: a polynucleotide encoding the capsid protein of a recombinant hybrid AAV in an expressed form. Also, the following is presented: an AAV-based vector particle for expression of the desired gene in muscle cells containing a nucleotide sequence corresponding to the capsid protein of the recombinant hybrid AAV.

EFFECT: invention expands the arsenal of agents for genetic therapy.

10 cl, 4 dwg, 3 ex

Similar patents RU2812279C2

Title Year Author Number
ENHANCEMENT OF TISSUE-SPECIFIC GENE DELIVERY BY CAPSID MODIFICATION 2019
  • Loiler, Scott Allen
RU2801217C2
VARIANT AAV, COMPOSITIONS AND METHODS, IN WHICH IT IS USED, AS WELL AS METHODS OF ITS APPLICATION FOR TRANSFER OF GENES INTO CELLS, ORGANS AND TISSUES 2014
  • Khaj Ketrin A.
  • Yazisioglu Mustafa N.
  • Angela Khaver
RU2697444C2
AAV VECTOR COMPOSITIONS AND METHODS FOR GENE TRANSFER TO CELLS, ORGANS AND TISSUES 2013
  • Khaj Katerine A.
  • Mingozzi Federiko
  • Sun Yunvej
  • Dzhonson Filip
RU2653444C2
MODIFIED CAPSID PROTEINS FOR IMPROVED DELIVERY OF PARVOVIRUS VECTORS 2016
  • Samulski, Richard Jude
  • Warischalk, Jayme
RU2751953C2
VARIANTS OF ACID ALPHA-GLUCOSIDASE AND THEIR USE 2017
  • Mingozzi, Federico
  • Ronzitti, Giuseppe
RU2780410C2
ADENO-ASSOCIATED VIRUS VECTOR 2015
  • Linden Ralph Michael
RU2743382C2
MODIFIED FACTOR IX, AS WELL AS COMPOSITIONS, METHODS AND OPTIONS FOR THE USE OF GENE TRANSFER INTO CELLS, ORGANS AND TISSUE 2016
  • Khaj Ketrin A.
  • Angela Khaver
RU2811445C2
MODIFIED FREEDREICH ATAXY GENES AND VECTORS FOR GENE THERAPY 2016
  • Samulski, Richard J.
RU2743792C2
RECOMBINANT VIRAL VECTORS WITH MODIFIED TROPISM AND WAYS OF THEIR USE FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS 2018
  • Kiratsus, Kristos
  • Merfi, Endryu Dzh.
  • Vang, Cheng
  • Sabin, Lea
RU2809246C2
NEW LIVER TARGETING ADENO-ASSOCIATED VIRAL VECTORS 2019
  • Colosi, Peter Cameron
  • Ramirez, Silvia
RU2793735C2

RU 2 812 279 C2

Authors

Rishar, Izabel

Zhikkel, Evelin

Mingotstsi, Federiko

Rontsitti, Dzhuzeppe

Vidal, Patris

Dates

2024-01-29Published

2019-04-04Filed